Ticagrelor (Brilinta®) has been in clinical use as an anti-clotting drug, but not for the treatment of cystic disease. Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited disease of the kidney, with a prevalence at birth ranging from 1 per 500 to 1000 people worldwide. ADPKD is caused by mutations in the PKD1 (85%) or PKD2 (15%) genes, which encode for polycystin-1 or polycystin-2 proteins, respectively. The hallmark of ADPKD is the formation of cysts in both kidneys, which gradually grow in size. Over the decades, new cysts form resulting in a decline of kidney function. By the age 55 years, about 50% of the ADPKD patients develop end-stage renal disease (ESRD), which requires dialysis therapy or renal transplantation.
There is no specific therapy for ADPKD. Its management is limited to control of high blood pressure, and symptomatic treatment of complications. Currently three different approaches are being tested to slow down the progression of cyst growth, but each has its own significant side effects.
Therefore, finding a treatment that can be sustained long-term without significant side effects is needed. The disclosed methods provide a new treatment for kidney diseases associated with elevated AVP (such as ADPKD) with a drug that is known to be safe for long term use in patients.